Jennifer Doudna, left, of the University of California, Berkeley, and French researcher Emmanuelle Charpentier, center, are allied against Feng Zhang, a scientist at the Broad Institute of MIT and Harvard, in a fight for the rights to the gene-editing tool Crispr.
Last month, Bayer AG opened the doors on a $335 million joint venture with Crispr Therapeutics to develop therapies using a new gene-editing tool. Later this year, rival Editas Medicine Inc. will move into larger digs as it, too, races ahead with a $200 million-plus effort to leverage the gene-editing tool into new drugs.
The system, known as Crispr-Cas9, is embroiled in a high-stakes patent dispute for control over what many scientists believe is a groundbreaking technology that serves as a kind of multipurpose molecular scissors that can cut DNA and make repairs and insert changes into genes. Crispr's power and versatility, they argue, open the door to new therapies that could potentially correct often intractable genetic diseases.
There have been other gene-editing tools before, and scientists are working on additional ones. But part of the excitement over the Crispr-Cas9 system is that it is easier to use than previous gene-editing technology. It also can target more than one gene at the same time, making it more efficient.
The different Crispr companies are hoping to develop treatments for diseases like hemophilia, cystic fibrosis and muscular dystrophy, among others. Doctors are planning to test Crispr therapies in cancer. Some scientists are even exploring the application of Crispr to revive extinct species like the woolly mammoth or to modify organs in pigs so they might someday be transplanted into humans.
Science is always a fraught, risky enterprise, but even by those standards this current fight to control Crispr-Cas9 is messy.
Jennifer Doudna of the University of California, Berkeley and Emmanuelle Charpentier, then of the University of Vienna, applied in 2013 for a patent for using Crispr-Cas9 to edit genes. So did Feng Zhang, a scientist at the Broad Institute of MIT and Harvard, in Cambridge, Mass., a few months later. But his patent was granted first, in 2014.
The U.S. Patent and Trademark Office is currently hearing a challenge by the Berkeley-Vienna group, which says it invented the gene editor first and should get the rights to the technology.
Scientists can use the gene-editing technology called Crispr-Cas9 to correct disease-causing mutations. Here's how it works.
A chunk of RNA is programmed to look for a specific problem segment of DNA. It is paired with a natural protein called Cas9 taken from bacteria, where it functions as a genetic scalpel.
Once inserted into a cell, the RNA/Cas9 combination looks for a DNA sequence that matches its RNA.
When it finds a match, the Cas9 cuts both strands of the DNA.
Repair enzymes can fill and seal the gap in the DNA with new genetic information to change the underlying genetic code.
This month, it got even more complicated when ToolGen, a Seoul-based biotech, said it was awarded a patent for Crispr-Cas9 gene editing in Korea. Seokjoong Kim, research director at ToolGen, said the company is also pursuing a claim with the USPTO, though an examiner hasn't yet deemed the claims patentable.
Many players laying claim to the key patent have launched companies to pursue research and licensing, creating a thicket of licensing options for biotechs and institutions to consider, depending on how they want to use the technology.
The Numbers : Read more about some of the companies that are racing to develop therapies using the Crispr-Cas9 gene-editing system, without waiting to see who wins the patent dispute.
Broad's Dr. Zhang is a co-founder of Editas Medicine. Dr. Charpentier, now of the Max Planck Institute for Infection Biology in Berlin, is a co-founder of Crispr Therapeutics, and Dr. Doudna, who helped found and then left Editas, is a co-founder of Caribou Biosciences Inc. and Intellia Therapeutics Inc. And there is a welter of other startups with rights to various uses and other technologies related to Crispr.
On Thursday, Monsanto Co. announced it had reached a deal with the Broad Institute to license the gene-editing tool for use in agriculture. The St. Louis, Mo.-based company said the license would deliver an array of crop improvements.
Patent experts say companies must decide whether to choose a side from among these, perhaps license from more than one to hedge their bets, or just use the technology and seek a license only when necessary.
Some legal experts said that with the rights in dispute, the risk of getting sued for patent infringement is low, and a company without cash for multiple licenses in the U.S. could be better off pushing ahead with its own research and waiting until the dispute is resolved.
At Fulcrum Therapeutics in Cambridge, Mass., which is developing therapies for diseases like Fragile X syndrome and a form of muscular dystrophy, CEO Robert Gould says its scientists didn't want to wait. Fulcrum was launched earlier this year with $55 million in financing from Third Rock Ventures LLC, also a founding investor in Editas.
A spokeswoman for Crispr Therapeutics says the company doesn't comment on the patent dispute. Executives and spokesmen for the other companies licensing out Crispr rights expressed confidence in their intellectual property and the ability for drug discovery to move forward.